Novartis Gene Therapies, Inc. (United States)

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Publications

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2.7K

Location

Bannockburn, United Kingdom

Also known as NGT · Novartis Gene Therapies · Novartis Gene Therapies, Inc.

Novartis Gene Therapies, Inc. (United States) is a company institution in Bannockburn, United Kingdom known for neurology, spinal muscular atrophy, and medicine.

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Top concepts

NeurologyMedicineSpinal Muscular AtrophyOnasemnogene Abeparvovec TherapyInfantile Spinal Muscular AtrophyPediatric Spinal Muscular AtrophyNeuroscienceNeurodegenerative DiseasesNeurodegenerative DisordersSpinal Muscular Atrophy Pharmacotherapy

Publications

Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy type 1 (STR1VE-EU): an open-label, single-arm, multicentre, phase 3 trial

Eugenio Mercuri, Francesco Muntoni, Giovanni Baranello et al. · The Lancet Neurology · 2021 · 256 citations

Medicine, Neurology, Neuroscience +12

Cost-Effectiveness of Newborn Screening for Spinal Muscular Atrophy in England

Diana Weidlich, Laurent Servais, Imran Kausar et al. · Neurology and Therapy · 2023 · 36 citations · Full text

Pharmacy, Pediatrics, Pharmacoeconomics +17

Patients with Spinal Muscular Atrophy Type 1 Achieve and Maintain Bulbar Function Following Onasemnogene Abeparvovec Treatment

Katlyn McGrattan, Richard D. Shell, Rebecca Hurst-Davis et al. · Journal of Neuromuscular Diseases · 2023 · 29 citations · Full text

Medicine, Neurology, Dysphagia +16

Using the ALSFRS-R in multicentre clinical trials for amyotrophic lateral sclerosis: potential limitations in current standard operating procedures

Jaap N.E. Bakers, Adriaan D. de Jongh, Tommy M. Bunte et al. · Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration · 2021 · 22 citations · Full text

Life Sciences, Neurology, Clinical Neuroscience +14

Onasemnogene abeparvovec preserves bulbar function in infants with presymptomatic spinal muscular atrophy: a post-hoc analysis of the SPR1NT trial

Richard D. Shell, Katlyn McGrattan, Rebecca Hurst-Davis et al. · Neuromuscular Disorders · 2023 · 16 citations · Full text

Medicine, Neurology, Neuroscience +13

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